Phase 1 Sickle Cell Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who have been diagnosed with sickle cell disease (specific types: HbSS, HbS/B0 thalassemia, HbSD, or HbSO) or transfusion-dependent beta-thalassemia, confirmed by genetic testing.
• People aged 13 to 40 years.
• People with sickle cell disease who have had at least two episodes of acute chest syndrome in the past two years, OR at least three severe pain episodes requiring hospital or clinic visits and intravenous pain medication in the past two years.
• People with beta-thalassemia who have needed large amounts of blood transfusions (at least 100 mL per kg of body weight per year, or at least 10 units per year) over the two years before joining.
• People whose blood cell counts (white blood cells and platelets) fall within specific ranges considered acceptable by the trial team.
• People whose heart, lungs, kidneys, and liver are functioning at a level that the trial team considers adequate (confirm with trial site for specific thresholds).
• People with sickle cell disease where a common medication called hydroxyurea has not worked well enough, caused side effects, or was not appropriate for their situation — or who are still taking hydroxyurea but continue to meet the disease severity criteria.
• People for whom no matched related bone marrow donor is available.
• People (or their parent or guardian, where applicable) who have signed the informed consent form.
• People willing to attend follow-up visits for 15 years after treatment.
• People who do not carry a specific genetic variation at a particular location in the genome known as rs114518452 (confirm with trial site).

Who may not be able to join:

• People with an uncontrolled or active infection, such as a current fever, an infection requiring intravenous antibiotics, or a serious fungal infection.
• People with an active cancer diagnosis.
• People experiencing a serious complication of their blood disorder that the trial investigators consider too risky for participation.
• People who have had major surgery within the past 30 days.
• People with medical, psychiatric, or personal circumstances that the treating doctor believes would make it difficult to follow the study requirements.
• People who cannot safely receive the preparatory medication (busulfan) used before the gene therapy.
• People who have previously had a bone marrow or stem cell transplant of any kind.
• People whose bone marrow sample shows signs of a pre-cancerous blood condition (myelodysplastic syndrome) or certain genetic mutations linked to blood cancers, detected through specialised testing.
• People with sickle cell disease who have severe narrowing or blockage of major blood vessels in the brain, or a condition called Moyamoya disease.
• People with sickle cell disease who are currently receiving regular blood transfusions specifically to prevent stroke (primary or secondary), or who previously had abnormal results on a brain blood flow test (transcranial Doppler) and were transitioned to hydroxyurea for stroke prevention.
• People with sickle cell disease who have had a stroke or any neurological event lasting more than 24 hours. (Note: silent strokes seen on imaging, where the person is not on a regular transfusion programme, are not automatically excluded — confirm with trial site.)
• People with very severe iron build-up in the body, if the trial investigators consider this level of iron overload too risky for participation.
• People who test positive for HIV, Hepatitis C (HCV), Hepatitis B (HBV), or HTLV.
• People with active hepatitis or significant liver scarring (moderate to severe fibrosis or cirrhosis) shown on a previous liver biopsy.
• People who have received another experimental drug or taken part in another clinical trial procedure within the past 90 days.
• People who are pregnant, breastfeeding, or not using adequate contraception. People who are able to have children must agree to use an acceptable form of birth control from the start of screening until at least six months after receiving the treatment.
• People carrying a specific genetic variation at a location in the genome identified as rs114518452 (this refers to having at least one copy of a particular DNA letter change at that site — confirm with trial site).
• People whom the trial investigators or transplant doctors determine are not suitable for the stem cell transplant process or other study procedures for any other reason.

Important: Always verify eligibility with the trial site directly before applying.